Problems and Purpose

Recent advances in science, particularly in molecular biology and genomics, mean that it will become increasingly possible to identify the underlying molecular mechanisms of disease. Developing diagnostic tests that indicate the molecular cause of a disease enables new treatments to be developed that can target diseases more precisely. Although these technologies are quite new, it is argued that they have the potential ability to predict which patients will respond to a given treatment.

This potential ability and being able to provide the exactly appropriate treatment has become known as ‘stratified’ medicine, indicating the division of patients into ‘strata’ of those who would be expected to respond to a particular medicine. 

The public dialogue project focused on identifying the human issues raised by stratified medicine, the implications for how it is delivered and what it will mean for individuals who will benefit from it and for those who will not be able to benefit from it. 

The objectives of the public dialogue were: 

• To discover the diversity of public opinion about stratified medicine as well as how best to explain what it involves, and which terms are least likely to cause confusion, misinterpretation or misunderstanding, so that stratified medicine and the issues it raises can be discussed effectively with patients, their families, and the general public
• To explore the possibilities of stratified medicine through a process that enables patients and members of the public to identify advantages and disadvantages that developers and healthcare providers may be overlooking, and to think creatively about ways to amplify the former and mitigate the latter  
• To identify what steps practitioners and other healthcare providers will have to take to communicate the complex information that patients and their families will need about the testing processes that stratified medicine requires, and the support that different strata of patients will require before, during and after treatment  
• To establish what sort of ethical framework and practical approaches to consent for trials will build patient and public confidence in and support for the sharing of the personal data necessary to ensure the effectiveness of stratified medicine. [1]  

Background History and Context

Even the best medicines are not equally effective in all patients. Disease processes and treatment choices can vary from person to person even though they may have similar symptoms. It is estimated that only 30% to 70% of patients respond positively to any particular drug. 

Stratified medicine has been summarised as identifying the right therapy for the right patient at the right time in the right dose. 

The introduction of stratified medicine would involve a number of key strategic shifts in the approach to diseases and their treatment. These have the potential to affect people in every area of healthcare – from investors in drug companies and researchers in their laboratories to general practitioners in their surgeries and patients in their homes. 

As the potential and implications of genomic medicine have become more apparent, the interest in stratified medicine has intensified since it began in around 2004. It offers the possibility of an evolution in healthcare that is based, increasingly, on early prediction and rapid prevention, rather than on later diagnosis and treatments that may come to seem, in retrospect, comparatively crude. The implications of the changes as a result of stratified medicine are not just potential improvements in healthcare, but also cultural shifts in our understanding of disease and the role of our health services in responding to it. 

Several Government bodies and leading charities are working together to accelerate the development and uptake of stratified medicine in the UK. Innovate UK (formerly the Technology Strategy Board), the Medical Research Council (MRC), Cancer Research UK (CRUK), Arthritis Research UK (ARUK), the Department of Health (DH), the Scottish Government Health Directorate (SGHD) and the National Institute for Health and Care Excellence (NICE) have formed a partnership to take forward the Stratified Medicine Innovation Platform (SMIP). Together, they will invest around £200 million over five years in the area of stratified medicine. 

Innovate UK works to stimulate innovation and economic growth in the UK. The UK is seen to have the potential to be a world leader in the development of stratified medicines due to the strength of its pharmaceutical and bioscience industries, its outstanding academic base, its single healthcare system (the NHS) and the work of many other organisations. Healthcare is the most highly funded priority area for Innovate UK. A part of that funding is being used to promote the development and uptake of stratified medicine with a range of partners through the SMIP. 

In 2011, members of the SMIP developed a roadmap that highlighted nine areas they saw as vital to support the uptake of stratified medicine. It was recognised that in many of these areas there were questions to be answered about how the public, patients and healthcare professionals would receive the proposed changes. The dialogue was designed to start to answer these questions. [1]

Organizing, Supporting, and Funding Entities

Cost of project: £207, 000 (Sciencewise-ERC co-funding £108,000)

The project was commissioned by Innovate UK (formerly the Technology Strategy Board), which stimulates technology enabled innovation in the areas which offer the greatest scope for boosting UK growth and productivity.

The project also received funding and support in the form of expertise from Sciencewise ERC. Following an open call, OPM and Dialogue by Design were contracted to deliver the project. Rhuari Bennett of 3KQ led the independent evaluation of the dialogue.  

Department of Health (DH) is the government department responsible for public health issues.

The Medical Research Council (MRC) is a publicly-funded organisation dedicated to improving human health.

Cancer Research UK is a leading cancer research and awareness charity based in the United Kingdom.

Arthritis Research UK is the leading authority on arthritis in the UK, conducting scientific and medical research into all types of arthritis and musculoskeletal conditions.

National Institute for Health and Clinical Excellence (NICE) provides independent, authoritative and evidence-based guidance on the most effective ways to prevent, diagnose and treat disease and ill health.

Scottish Government Health and Community Care is responsible for NHS Scotland and for the development and implementation of health and community care policy [2]

Sciencewise-ERC is a Department for Business, Innovation and Skills funded programme to bring scientists, government and the public together to explore the impact of science and technology in our lives. It helps Government departments and agencies commission and use public dialogue to inform policy making, involving science and technology issues. Its core aim is to develop the capacity of Government to carry out good dialogue, to gather and disseminate good practice, have successful two-way communications with the public and other stakeholders, and to embed the principles of good dialogue into internal Government processes.

Participant Recruitment and Selection

• Total public participants involved: 152
• Public workshops: 51
• Targeted workshops: 38
• Self-facilitated workshops: 63
• Total stakeholders involved: 40 at stakeholder workshop
• Total experts: 7 across the public workshops

Governance and Oversight membership

An Oversight Group (OG) was set up for the project, consisting of a range of people with specialist knowledge who could help ensure the process was fairly, objectively and sensitively conducted. This group included experts from medical research and bio-ethics, alongside representation from medical charities, patient groups and the pharmaceutical industry. The full membership is available in the Evaluation report.[3]

• Public Workshops: 2 locations, met twice; 24-27 participants per session recruited via purposive sampling; incentives: £40 each day 1, £60 each day 2
• Targets Workshops: 4 groups, met twice; 5-15 participants per session recruited via intermediary groups; incentives: £25 session 1, £40 session 2
• Self-Facilitated Workshops: 7 twice, met once; 5-8 participants recruited as pre-existing groups; incentives: £80 per group
• Stakeholder Workshop: met once; 40 participants, self-selected after receiving a board invitation; no financial incentive

Methods and Tools Used

The engagement process included facilitated public and stakeholder workshops as well as self-facilitated discussions, the latter of which was employed to explore how people interpreted stratified medicine outside of the more managed process of the main dialogue, as they might encounter it in the real world. 

What Went On: Process, Interaction, and Participation

Public Workshops

A team of at least four facilitators and/or note-takers from the delivery contractor staffed these events. Additional specialists observed the events and assisted by answering questions when appropriate. Participants were taken through a schedule of activities that enabled them to learn about stratified medicine, ask questions, discuss the issues, and also give their views.

Targeted Workshops

Each targeted group was invited to meet twice for about 2 hours each time, with time in-between to consider the issues they had discussed. The exception was the medical students whose schedule only allowed for one session.

The delivery contractor facilitated these sessions, and there was no specialist support apart from at the session for medical students (where a member of the Oversight Group attended and helped to answer questions). More flexibility was allowed as to which topics each group covered. This enabled each group to focus on what it had most to say about. For example, patient groups spent more time on care implications than the public.

Self-facilitated groups

The self-facilitated groups were provided with the same materials as the other groups but one group member was asked to facilitate the discussion after a briefing with the delivery contractor. There was no facilitator from the delivery contractor, and no specialist to answer questions. The aim was to explore how people interpreted stratified medicine outside of the more managed process of the main dialogue, as they might encounter it in the real world.

The self-facilitated groups had the shortest discussions, just one session of around two hours. This reflected both the more limited nature of debate that might naturally occur in an everyday setting. The delivery contractor followed up each session with a telephone interview with the group’s facilitator.  

Stakeholder workshop

After the three strands of dialogue above were complete, the findings were drawn together into a headline report, and discussed at a stakeholder workshop. This workshop aimed to involve stakeholders in reviewing the findings and exploring the implications for the future development of stratified medicine. Invitations were sent by the Board to over 100 organisations across industry, academia, government and the third sector. Around 40 stakeholders attended, in addition to representatives from the Board and project Oversight Group and two public participants from earlier stages of the dialogue (50 people in total). The workshop was a full day event, facilitated by the delivery contractor. [3]  

Key Messages from the Public

The participants identified a number of challenges, which can be grouped into four main themes: 

1. Definition and communication. Here, the challenges are: 

• having a clear, consistent definition of stratified medicine  
• presenting a realistic picture of stratified medicine, its pros and cons  
• continuing to engage the public and patients.

2. Implications for patients and care. Here the challenges are to:  

• support patients to make sound treatment decisions  
• support patients for whom there is no current treatment  
• provide the right facilities and training for healthcare professionals.  

3. Social issues and consequences. In this area, the challenges are to: 

• understand and mitigate any implications for equality  
• define the role of the private sector in developing stratified medicine  
• develop understanding of the costs/benefits of stratified medicine.  

4. Research, testing and data sharing. Here the challenges are to:  

• give research participants a choice about who uses their data and how it is used  
• reconcile the role and perception of the medical research industry  
• engage the public in regulation on data sharing. 

Influence, Outcomes, and Effects

The reports on the dialogue acknowledged limitations at the time of their publication in assessing impact. [1] [2] However, the case study observed the following influence on policy and policy makers.

Innovate UK has disseminated the dialogue results and been able to identify various immediate impacts of the dialogue. It has already changed the way that the Innovate UK staff involved communicate. In particular, they use the term ‘stratified medicine’ less and talk more about how it works and use other phrases such as ‘personalised’ or ‘tailored’. In addition, they stress that it is an evolution of established practice rather than a new concept. Innovate UK also intends to take account of the sensitivity to ethnicity identified in the dialogue and to ensure the focus is on increasing access to appropriate treatments. Further insights from the dialogue that were useful to Innovate UK included the differing levels of trust the public had in different institutions (Government and industry), and views on the use and sharing of data. 

The process also influenced the views of Innovate UK and stakeholders on working with the public in the future. Innovate UK reported that “Our colleagues are now talking about the merits of public dialogue on other topics” and an Oversight Group member commented that Innovate UK "recognised that the public do have interesting things to say. It wasn’t too frightening”.[1] According to another Oversight Group member, the dialogue provided “Confirmation that people are capable of making sense of complex information and, given time, that they can do it and come up with sensible opinions.”[1]

Analysis and Lessons Learned

What worked especially well 

Overall, this dialogue was very successful. The following five elements of the project worked particularly well. 

1. Setting up an Oversight Group before the contractor was appointed

The Oversight Group was involved closely in framing the original purpose of the dialogue, which added to the sense that stakeholder interests were being met. The Oversight Group brought a range of perspectives to bear on the process at every stage, including on the information provided for the public, and added to the credibility of the project and its results. 

2. Clear objectives, appropriate methods and good organisation

The clarity of the objectives, clearly communicated, and the design and delivery of the process overall, worked very well throughout. 

3. Sufficient time and an initial scoping review

The dialogue project had a good amount of time available – 10 months from the appointment of contractors (early June 2013) to closure (end March 2014). This allowed time to discuss things thoroughly among the delivery team, Innovate UK and the Oversight Group. It allowed the delivery contractor to hold a public pilot of the materials and make subsequent improvements, and also to try out slightly different processes via the targeted groups and self-facilitated groups. The initial scoping review clearly set out in one place the technical issues for the Oversight Group, helped frame the whole dialogue process, accelerated materials development and immersed the facilitator team in the technical content from the start. 

4. Targeting groups that were likely to be more affected

There was clear reason to suspect that some sections of the public could be more affected by stratified medicine than others. The inclusion of workshops for patients (young and adult), medical students, black and minority ethnic (BME), and patient support groups helped with triangulating the results and building the credibility of the results overall. These could not have replaced the purposively sampled public sessions, but were useful as an addition to them. 

5. Stakeholder workshop at the end to discuss the results

The inclusion of a stakeholder workshop after the dialogue events had been completed was very useful in ‘spreading the word’ about the dialogue’s existence and results, and building wider stakeholder buy-in to the dialogue process. 

What worked less well

The self-facilitated groups were less successful. These discussions focused on pre-existing groups, such as patient support groups and youth groups, and were a useful, but challenging, method. A volunteer from each group, who was briefed in advance by the delivery contractor, facilitated each discussion and took notes. 

The Oversight Group valued this strand as it focused attention on sectors of the public that may not otherwise have been included in any numbers in the project (particularly BME communities). However, it was quite difficult to persuade a range of groups to hold a discussion and it took a while to achieve even the seven groups that did meet. The delivery contractor suggested that the following things helped or may help in future: 

• If possible, invite the volunteer facilitators to the public events so they are well briefed and immersed in the technical content themselves.
• Target groups of people who have a clear interest in the benefits and implications of the technologies under discussion. In this case, patient groups offered the best return.
• Be ready to offer a financial incentive that is comparable to the public workshops. 
• It is hard for volunteer facilitators to capture a full and accurate set of notes. Following up with a debrief interview soon after the event to hear about discussions can help make the data more robust. [1]  

See Also

Sciencewise

References

[1] Sciencewise (2014) “Case Study: Developing Stratified Medicine”, Sciencewise

[2] Sciencewise (2017) “Developing Stratified Medicine” [ONLINE] Available at: https://webarchive.nationalarchives.gov.uk/20170110132757tf_/http://www.sciencewise-erc.org.uk/cms/developing-stratified-medicine/

[3] Bennett, R (2014) “Evaluation of a Public Dialogue on Stratified Medicine”, 3KQ, June 2014

External Links

https://sciencewise.org.uk/ 

Notes